Rare Diseases

When you conduct clinical trials involving rare diseases, you face a unique set of medical, scientific, and operational challenges. PRA is an ideal partner to guide your rare disease clinical trials. Having conducted 189 trials over the last 5 years in this area, we possess a clear understanding of the challenges and critical success factors necessary to successfully develop drugs for rare disease patients. Our relevant experience encompasses Phase I-IV studies in 11 therapeutic areas and a wide range of indications. We have contributed to the successful market approvals of 14 drugs to treat rare indications.

PRA HAS MANAGED 189 PHASE I-IV RARE DISEASE CLINICAL STUDIES WITH 37,500+ PATIENTS AND 9,500+ SITES ACROSS A VARIETY OF THERAPEUTIC INDICATIONS. WE HAVE ALSO CONTRIBUTED TO THE APPROVAL OF 14 RARE DISEASE DRUGS.

PRA’s Approach to Rare Disease Development Programs

Rare disease trials require a customized approach based on the disease specifics and study goals. Through our experience, PRA has developed an effective planning and execution process that includes:

 

Therapeutic_Expertise_Rare_Diseases_Advocacy_Group_Engagement_Graphic_White

VALUE-ADDED SOLUTIONS in Rare Diseases

PRA has identified several critical success factors for rare disease studies:

  • PRA’s evidence-based methodology provides access to proprietary databases, feasibility studies and key investigator relationships to enhance patient recruitment.
  • Strong partnerships with relevant patient advocacy groups create stronger study awareness and support.
  • Quality subject support materials demonstrate the benefits of participation to subjects, leading to increased motivation to enroll.
  • A core set of study-branded tools help sites identify potential patients as well as educate them about the study and its requirements.
  • PRA’s Patient Access and Retention Services (PARS) team partners with best-in-class patient recruitment organizations to develop customized programs.